Treatment Trials

17 Clinical Trials for Various Conditions

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COMPLETED
Caprylic Triglyceride for Treatment of Cognitive Impairments in Multiple Sclerosis
Description

Background/Rationale: Cognitive problems are a common symptom in individuals with Multiple Sclerosis (MS). Treatment options are limited, and there is a pressing need for new interventions to treat MS-related cognitive impairment. Glucose (a type of sugar) is used to fuel the cells of the healthy brain. For people with neurological conditions such as MS, glucose is not converted into energy as efficiently as it would be in a healthy brain, which can lead to a decrease in cognitive function. Caprylic Triglyceride may work to bypass this problem by providing an alternative energy source that is metabolized in the liver and used by the brain. Objective: To evaluate the therapeutic effects of 90 days of caprylic triglyceride on cognitive impairment in multiple sclerosis. Design: Randomized, double blinded, placebo controlled trial of 158 subjects. Outcome: Change in Total Learning (Trials 1-5) on the California Verbal Learning Test-2nd Edition-(CVLT-II) AND Change in Symbol Digit Modalities Test (SDMT) (at day 90

RECRUITING
A Study to Assess the Safety and Clinical Activity of Azer-cel in Participants With B-cell Mediated Autoimmune Disorders
Description

The main objective of the study is to determine the recommended phase 2 dose (RP2D) of Azercabtagene zapreleucel (azer-cel).

COMPLETED
An Observational Biomarker Study in Multiple Sclerosis (MS) Patients
Description

All males and females between 18-70 years of age regardless of their race and ethnicity with a confirmed diagnosis of Primary Progressive Multiple Sclerosis (PPMS), Secondary Progressive Multiple Sclerosis (SPMS), and Relapse Remitting Multiple Sclerosis (RRMS) are invited to participate in this Observational study being conducted across four sites in the US. Since this is an observational study no medication/drug or treatment will be given to the participants. The investigator will be collecting information about the participant's MS disease, its progression, current medications, radiographic scans, and blood samples. This will help the investigator evaluate the biomarkers and new treatment options to better understand the MS disease process.

RECRUITING
Assessing Changes in Multi-parametric MRI in MS Patients Taking Clemastine Fumarate as a Myelin Repair Therapy
Description

The clinical trial is intended to assess for clinical evidence of Clemastine Fumarate as a myelin repair therapy in patients with chronic inflammatory injury-causing demyelination as measured by multi-parametric MRI assessments. No reparative therapies exist for the treatment of multiple sclerosis. Clemastine fumarate was identified along with a series of other antimuscarinic medications as a potential remyelinating agent using the micropillar screen (BIMA) developed at the University of California, San Francisco (UCSF). Following in vivo validation, an FDA IND exemption was granted to investigate clemastine for the treatment of multiple sclerosis in the context of chronic optic neuropathy. That pilot study was recently completed and is the first randomized control trial documenting efficacy for a putative remyelinating agent for the treatment of MS. The preselected primary efficacy endpoint (visual evoked potential) was met and a strong trend to benefit was seen for the principal secondary endpoint assessing function (low contrast visual acuity). That trial number was 13-11577. This study seeks to follow up on that study and examine clemastine fumarate's protective and reparative effects in the context of chronic demyelinating brain lesions as imaged by multi-parametric MRI assessments. The investigators will be assessing the effects of clemastine fumarate as a remyelinating therapy and assessing its effect on MRI metrics of chronic lesions found in patients with a confirmed diagnosis of relapsing-remitting multiple sclerosis. In addition to using conventional multi-parametric MRI assessments, this study will also evaluate a new MRI technique called Ultrashort Echo Time (UTE) MRI to assess the effects of clemastine fumarate as a remyelinating therapy of chronic lesions found in patients with a confirmed diagnosis of relapsing-remitting multiple sclerosis and compare it to the other assessments.

RECRUITING
MS-ResearchBiomarkerS
Description

This study is being conducted to investigate risk factors for disability progression in Multiple Sclerosis and related disorders (MSRD). The primary goal is to assess whether combining information from visual assessment, blood markers, as well as historical and ongoing longitudinal MRIs of the brain, orbit (the part of the skull where eyes are located), and/or spinal cord can predict changes in quantitative disability measures related to MSRD and neurological disease.

ACTIVE_NOT_RECRUITING
Intrathecal Administration of DUOC-01 in Adults With Primary Progressive Multiple Sclerosis
Description

This study is a prospective Phase 1a open-label single- center trial. It will assess the safety of intrathecal administration of DUOC-01 cells to adults with Primary Progressive Multiple Sclerosis (PPMS). DUOC-01 is a population of cells expanded from donated human umbilical cord blood cells and is intended for treatment of neurodegenerative and demyelinating diseases. There will be approximately 20 participants enrolled. Exploratory objectives include changes in MS assessment scores, changes in brain MRI findings, and changes in blood biomarkers.

UNKNOWN
Novel Assessment of Synaptic Density in Progressive MS
Description

The investigators propose to use the novel SV2a-PET ligand, \[F-18\]SDM-8 to assess synaptic density in progressive MS (including primary progressive multiple sclerosis (PPMS) and secondary progressive multiple sclerosis (SPMS)) as compared to relapsing-remitting multiple sclerosis (RRMS) patients and healthy controls, given its improved imaging characteristics and potential for large scale applicability. The specific aims of the study are: Aim 1: To compare the cortical and subcortical grey matter synaptic density in progressive MS patients, patients with relapsing-remitting MS, and healthy subjects, using a novel \[F-18\] labeled synaptic density PET ligand, \[F-18\]SDM8, also known as \[F-18\]SynvesT-1. Aim 2: To compare the relationship of synaptic density PET and standard 3T MRI measures including global and regional brain atrophy and lesion load with clinical measures of physical disability, cognitive impairment, fatigue and depression in MS patients. Aim 3: To assess the relationship of synaptic density PET with serum neurofilament light chain (NfL) and with serum measurements of inflammatory markers, IL-1β, TNF-α, IL-6, MCP-1 (Monocyte Chemoattractant Protein-1) and MIF-1 (Macrophage Migration Inhibitory Factor-1).

ACTIVE_NOT_RECRUITING
Evaluation of a Theory-Driven Manualized Approach to Improving New Learning and Memory in MS
Description

The purpose of this research study is to investigate the effectiveness of a memory enhancement technique in persons with Multiple Sclerosis.

COMPLETED
Open-label Study of Liothyronine in MS
Description

This study will evaluate the safety and tolerability of synthetic T3, liothyronine. It will establish if there are changes in MS symptoms and if there is a positive effect on markers of neuronal health.

TERMINATED
ACTH in Progressive Forms of MS
Description

This is a phase II, randomized, double-blind, placebo-controlled, multi-center study to evaluate the safety, tolerability, and efficacy of adrenocorticotropic hormone (ACTH, Acthar gel) administered as a pulsed regimen consisting of injections on three consecutive days per month in patients with progressive forms of Multiple Sclerosis (MS). Patients will be randomly assigned to either an ACTH arm or a placebo arm. The main hypotheses are that 1) pulsed ACTH will be safe and well-tolerated, and 2) pulsed ACTH will slow progression of clinical and paraclinical measures of MS progression compared to placebo.

UNKNOWN
Estriol Treatment in Multiple Sclerosis (MS): Effect on Cognition
Description

Approximately 50% of people diagnosed with Multiple Sclerosis (MS) will develop problems with cognition. Currently, there are no FDA-approved treatments targeting cognitive function in Multiple Sclerosis. This trial will ascertain whether treatment with an estrogen pill, used in combination with standard MS anti-inflammatory drugs, can improve cognitive testing as compared to treatment with a placebo pill in combination with standard anti-inflammatory drugs in women with MS.

COMPLETED
Clinical Trial of Idebenone in Primary Progressive Multiple Sclerosis (IPPoMS)
Description

Background: * Multiple sclerosis (MS) is an inflammatory disorder of the central nervous system that progressively weakens and destroys the pathways of the nervous system. About 10 percent to 15 percent of patients develop primary-progressive MS (PP-MS), characterized by progressive accumulation of disability from the disease onset, without any marked improvements or relapses. There are currently no effective treatments for PP-MS. * Idebenone is a manmade drug that is similar to a naturally occurring compound known as coenzyme Q10, a common dietary supplement. Research data suggest that idebenone may be able to limit demyelination and death of brain cells and thereby slow or halt the progression of neurological dysfunction such as that occurring in MS. Objectives: - To evaluate the safety and effectiveness of using idebenone to treat primary progressive MS. Eligibility: - Individuals between 18 and 65 years of age who have been diagnosed with primary progressive multiple sclerosis. Design: * The study will last 3 years and will be divided into two parts: a 1-year pretreatment baseline and 2 years of treatment with either idebenone or a placebo. * Pre-treatment study: approximately 5 clinic visits over 1 year. * Visit 1: Comprehensive medical history and neurological examination, with brain scans and neurological tests. * Visit 2: Magnetic resonance imaging (MRI) scan of the spine and lymphocytapheresis (withdrawal of white blood cells for testing). * Visit 3: Lumbar puncture. * Visit 4: Skin biopsy. * Visit 5: Repeat MRI of the brain and spinal cord, as well as neurological tests; these tests will be scheduled over 2 days. * After the five pretreatment visits, patients will receive a 6-month supply of study medication (either idebenone or a placebo) to take three times a day with food * Patients will continue to have regular followup clinic visits with brain MRI scans, blood tests, and other evaluations of brain and nervous system function. Randomly selected participants will have additional MRI scans for further safety precautions.

ACTIVE_NOT_RECRUITING
KYSA-7: A Study of Anti-CD19 CAR T-Cell Therapy, in Subjects With Refractory Primary and Secondary Progressive Multiple Sclerosis
Description

A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell (CD19 CAR T) Therapy, in Subjects with Refractory Primary and Secondary Progressive Multiple Sclerosis

TERMINATED
Phase 1/2 Study to Evaluate the Safety and Efficacy of ATA188 in Subjects With Progressive Multiple Sclerosis
Description

The purpose of this study is to evaluate the safety and tolerability of ATA188 as a monotherapy in Parts 1 and 2, to determine the recommended Part 2 dose (RP2D) of ATA188 as monotherapy in Part 1, and to evaluate the effect of ATA188 treatment on clinical disability, as assessed by confirmed Expanded Disability Status Scale (EDSS) improvement at 12 months in Part 2 in participants with progressive forms of multiple sclerosis (MS) (primary progressive multiple sclerosis \[PPMS\] and secondary progressive multiple sclerosis \[SPMS\]).

COMPLETED
Video Game-based Therapy for Arm Weakness In Progressive Multiple Sclerosis
Description

Participants will receive intensive therapy through a video game in their own homes for 1 month. The game is operated by movements of the body (no controller needed) and acts like a virtual therapist. Participants will agree to play the game for 30 hours. They will also receive 4 home visits from a therapist designed to increase use of their arm for daily activities that are important to them. Participants will also receive a smart watch that will measure use of their weaker arm and alert them when their arm is not participating in activities. Arm strength and function will be measured before and after the therapy program.

SUSPENDED
IDP-023 g-NK Cells Plus Ocrelizumab in Patients With Progressive Multiple Sclerosis
Description

This is an open label, Phase 1b, multiple ascending dose, and dose-expansion study of IDP-023 administered in combination with interleukin-2 (IL-2) and ocrelizumab to evaluate the safety, tolerability, and biologic activity on autoreactive immune cells in patients with refractory progressive multiple sclerosis.

RECRUITING
Phenotype, Genotype and Biomarkers 2
Description

The purpose of this study is to learn more about amyotrophic lateral sclerosis (ALS) and other related neurodegenerative diseases, including frontotemporal dementia (FTD), primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA) and multisystem proteinopathy (MSP). More precisely, the investigator wants to identify the links that exist between the disease phenotype (phenotype refers to observable signs and symptoms) and the disease genotype (genotype refers to your genetic information). The investigator also wants to identify biomarkers of ALS and related diseases.